Working with Medicines Regulators

This month, PSC Support was invited by the UK medicines regulator (the MHRA) to join other rare disease charities in shaping their new research plans for rare conditions.

 

Research into rare diseases like PSC is often more complicated than for common conditions. For example, during COVID-19, research moved quickly because there were so many people available to take part in studies, and systems were adapted to speed up the process.

 

Rare diseases don’t have those advantages, which can make progress slower. The MHRA is listening to charities like ours to explore how things can be improved. We hope this will lead to more efficient ways of developing medicines for rare conditions like PSC.

 

On a related note, I’ve also been appointed as a Lay Member of the MHRA’s Innovative Licensing and Access Pathway (ILAP) Delivery Group. This is a group that brings together key UK organisations (NICE, NHS England, the Scottish Medicines Consortium and others) to work with medicine developers. Usually, each organisation asks for different types of evidence to prove a new medicine is effective or value for money, which can add time and cost. ILAP helps streamline this, offering a clearer, joined-up route for promising new medicines.

 

My role as a Lay Member is to provide the ‘patient and public’ perspective when the group looks at whether new medicines should be considered for this pathway. While I won’t be involved in any decisions about PSC medicines directly, this role is strategic for PSC Support.  It allows us to contribute to medicine development in the UK, build PSC Support’s understanding of regulatory processes and it strengthens our ability to advocate for PSC medicines as they come along.

 

UK Liver Research Conference

Next week, we’ll be heading to Belfast for the British Association for the Study of the Liver’s annual meeting. The conference is an opportunity for clinicians to meet and learn about the latest developments in liver disease research and care.

 

We’re particularly excited because PSC is firmly on the agenda this year. Highlights include a presentation from Professor Trauner on emerging PSC treatments (in May, he shared results from a large Phase 3 clinical trial), a session on how best to monitor people with IBD and PSC, and another on recurrent PSC (when the condition returns after a liver transplant).

 

It’s fantastic to see PSC research and knowledge being shared with a wider audience of doctors and nurses. For a long time, PSC has been in the shadows so raising awareness in this way is an important step forward.

 

Improving Quality of Life

Last week, Mark Chatterley and I travelled to Poland to represent PSC patients at an international workshop to bring together researchers working on measuring quality of life in PSC.

 

We urgently need a way to measure quality of life in PSC that captures the true impact of the disease. Mark’s outstanding presentation of his personal story conveyed the long-lasting and far reaching effects of PSC, showing the disease is so much more than medical test results. Find out more here.

 

Burden of Illness Survey

We’ve also been working with Mirum to help capture what it really means to live with PSC. This includes not just symptoms, but also the emotional, social and financial impact of the disease. These insights are so important for researchers and companies developing future treatments, ensuring they reflect the realities of daily life with PSC. If you have itch, please find out more here and complete the survey.

 

Hope for New PSC Studies

Over the summer, I’ve been collaborating with researchers on three major funding applications for new PSC studies, both in the UK and internationally. While I can’t share details just yet, please keep your fingers crossed. Two more applications are already lined up for October, and we’ll be involving members of our brilliant Patient Panel pool.

 

If you’d like to join the Patient Panel pool, it’s a great way to learn more about PSC research and actually influence what is planned. You can sign up here.

 

Our Growing Research Team

Thank you to everyone who responded to our recent call for research volunteers! Four fantastic people will be joining me and Maria Richardson from next month, and we’re also delighted that Mark will now be dedicating part of his time each week to research activities.

 

Get in Touch

Do drop me an email if you have any questions:

Martine Walmsley

PSC Support Head of Research Strategy
ERN RARE-LIVER Management Board and PSC Working Group Co-Lead

martine@pscsupport.org.uk